Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
HaploSCD
Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)
2 other identifiers
interventional
21
1 country
6
Brief Summary
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients. Funding Source - FDA OOPD
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jan 2012
Longer than P75 for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 19, 2011
CompletedFirst Posted
Study publicly available on registry
October 28, 2011
CompletedStudy Start
First participant enrolled
January 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2026
ExpectedAugust 8, 2025
August 1, 2025
13.9 years
October 19, 2011
August 7, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Treatment related events
Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease
1 year
Secondary Outcomes (3)
neurological/neurocognitive status
2 years
Pulmonary/pulmonary vascular status
2 years
Health-related quality of life
4 years
Study Arms (1)
Haplo Stem Cell Transplantation
EXPERIMENTALCD34 selected T-cell depleted allogeneic SCT
Interventions
Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0
Eligibility Criteria
You may qualify if:
- Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
- Patients must demonstrate one or more of the following Sickle Cell Disease Complications
- Clinically significant neurologic event (stroke) or any neurologic deficit lasting \>24 hours that is accompanied by an infarct on cerebral MRI
- Minimum of two episodes of acute chest syndrome.
- Recurrent painful events (at least 3 in the 2 years prior to enrollment).
- Abnormal TCD study requiring starting on chronic transfusion therapy.
- At least one silent infarct lesion on a MRI scan of the head.
- A familial haploidentical donor without homozygous sickle cell disease
- Adequate organ function (renal, liver, cardiac and pulmonary function)
- Karnofsky or Lansky (age appropriate) Performance Score ≥50%
- Liver biopsy is optional to assess for iron overload in chronically transfused patients.
You may not qualify if:
- Females who are pregnant or breast-feeding
- SCD Patients with documented uncontrolled infection
- SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
- Demonstrated lack of compliance with medical care.
- Clinically significant fibrosis or cirrhosis of the liver
- Previously received a HSCT
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- New York Medical Collegelead
- UCSF Benioff Children's Hospital Oaklandcollaborator
- Medical College of Wisconsincollaborator
- Washington University School of Medicinecollaborator
- Tufts Medical Centercollaborator
- University of California, San Franciscocollaborator
- University of California, Los Angelescollaborator
- Miltenyi Biomedicine GmbHcollaborator
- Ann & Robert H Lurie Children's Hospital of Chicagocollaborator
Study Sites (6)
University of California Los Angeles (UCLA)
Los Angeles, California, 90095, United States
Children's Hospital and Research Center Oakland
Oakland, California, 94609, United States
Lurie Children's Hospital
Chicago, Illinois, 60611-2605, United States
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
New York Medical College
Valhalla, New York, 10595, United States
Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (2)
Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS. Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial. Front Neurol. 2024 May 22;15:1263373. doi: 10.3389/fneur.2024.1263373. eCollection 2024.
PMID: 38841694DERIVEDParsons SK, Rodday AM, Weidner RA, Morris E, Braniecki S, Shenoy S, Talano JA, Moore TB, Panarella A, Flower A, Milner J, Fabricatore S, Mahanti H, van de Ven C, Shi Q, Cairo MS. Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant. Bone Marrow Transplant. 2022 Apr;57(4):586-592. doi: 10.1038/s41409-022-01584-y. Epub 2022 Feb 2.
PMID: 35110690DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Mitchell S Cairo, MD
New York Medical College
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
October 19, 2011
First Posted
October 28, 2011
Study Start
January 1, 2012
Primary Completion
December 1, 2025
Study Completion (Estimated)
December 1, 2026
Last Updated
August 8, 2025
Record last verified: 2025-08